Utilizing Response- and Biology-Based Risk Factors to Guide Therapy in Patients with Non-High-Risk Neuroblastoma

This phase III trial studies how well response and biology-based risk factor-guided therapy works in treating younger patients with non-high risk neuroblastoma. Sometimes a tumor may not need treatment until it progresses. In this case, observation may be sufficient. Measuring biomarkers in tumor cells may help plan when effective treatment is necessary and what the best treatment is. Response and biology-based risk factor-guided therapy may be effective in treating patients with non-high risk neuroblastoma and may help to avoid some of the risks and side effects related to standard treatment.

Study details

Group A (clinical observation)

• Patients undergo clinical observation for 96 weeks in the absence of disease progression.

Group B (clinical observation, first-line chemotherapy)

• Patients undergo clinical observation for 3 years in the absence of disease progression.
• Upon disease progression, patients undergo surgery or receive first-line chemotherapy comprising carboplatin IV over 1 hour on day 1 (courses 1, 2, 4, 6, and 7), etoposide IV over 1 hour on days 1-3 (courses 1, 3, 4, 5, and 7), cyclophosphamide IV over 1 hour on day 1 (courses 2, 3, 5, 6, and 8), and doxorubicin hydrochloride IV over 15 minutes on day 1 (courses 2, 4, 6 and 8).
• Treatment with chemotherapy repeats every 21 days for 2-8 courses in the absence of disease progression or unacceptable toxicity. Once a PR or better is achieved, patients undergo clinical observation for 3 years.

Group C (clinical observation, first-line chemotherapy)

• Patients at high risk for deterioration and a poor outcome immediately receive first-line chemotherapy as in Group B.
• All other patients undergo clinical observation for 3 years in the absence of disease progression.
• Upon disease progression, patients receive first-line chemotherapy as in Group B.
• Once a PR or better is achieved, patients undergo clinical observation for 3 years.

Eligibility

• Age < 12 months of age at diagnosis with INRG stage L1; or
• Age < 18 months of age at diagnosis with INRG stage L2 or stage Ms neuroblastoma/ganglioneuroblastoma
• Enrollment on ANBL00B1 is required for all newly diagnosed patients
• Patients must have newly diagnosed v-myc avian myelocytomatosis viral oncogene neuroblastoma derived homolog (MYCN) non-amplified neuroblastoma or MYCN non-amplified ganglioneuroblastoma verified by histology

Contact

For questions about study details and eligiblity criteria, please contact Ashley Bayne:

• 352-294-8745
• abayne@ufl.edu